GC Biopharma announced on Tuesday that the U.S. FDA has granted Fast Track Designation for GC1130A, a treatment for Sanfilippo syndrome type A (MPS IIIA) that it has been co-developing with Novel Pharma.

The fast-track designation follows the FDA's clearance of the Phase I investigational new drug (IND) application for GC1130A last month and is expected to further accelerate the development of GC1130A.

Sanfilippo syndrome (type A) is a rare genetic disorder that causes central nervous system damage through the accumulation of heparan sulfate, leading to progressive neurological decline. Without treatment, patients face life-threatening complications by the age of 15.

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